Research Project

Project Title:	RNA therapy in cystinosis
Project Type:	Translational research
Disease group(s):	Tubulopathies, Metabolic & stone disorders
Project Summary:	This research project investigates the potential of CTNS mRNA packed in lipid nanoparticles (LNP) to correct cystinosis phenotype, and in particular renal Fanconi syndrome. To this end we use proximal tubular cell models derived from patients with cystinosis, isogenic cell CTNS knock-out cell lines in 2D and 3D coculture, and animal models of different complexity (fish and rat). While proof-of-principle studies show the feasibility of this approach (Bondue et al. Sci Rep 2024), bypassing the liver and targeting CTNS-LNP to the proximal tubules is a new research challenge.
Lead principal investigator(s):	Elena Levtchenko, Amsterdam
Co-investigator(s):	Tjessa Bondue, Leuven
Project Period:	01/2023 - 01/2026
Sponsors:	Non-profit foundation, Local resources